In the summer of 2012, the Doudna Lab at the University of California, Berkeley published an article announcing, “Programmable DNA Scissors Found for Bacterial Immune System.” The discovery – sandwiched between a much-anticipated Facebook IPO and a landmark Supreme Court ruling on healthcare coverage – entered the world largely under the public’s radar. But it wouldn’t stay there for long. Just a few years later, this new technology for genome editing was named “Breakthrough of the Year” by Science Magazine, gracing the mainstream media covers of both Time and National Geographic in 2015, and earning co-authors Jennifer Doudna and Emmanuelle Charpentier the Nobel Prize for Chemistry in 2020.
Today, therapies leveraging CRISPR-Cas9 technology have been approved by the European Medicines Agency and the FDA, making clinical gene editing a reality. The first example of such a therapy came with the recent FDA approval of Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The indications of this treatment are sickle cell disease (SCD) and beta thalassemia.
Credit: Author supplied
In the years to come, it will be exciting to watch where CRISPR technology goes next, what other indications see breakthroughs, and how further emerging technologies play a key role. Nearly 700 cell and gene therapies (CGT) are in development across the US with approximately 30 products already approved by the FDA. Success in leveraging CRISPR in this area will encourage greater investment in gene editing and greater investment in the creation of these advanced therapies. It is even predicted that 2024 could be a “breakout year” for gene therapy, and up to 17 gene therapies could be approved this year in the US and EU.
Conditions like SCD, where only one genetic mutation must be corrected, will continue to be strong candidates for CRISPR-based treatments. For other indications, the initial success of CRISPR in SCD and beta thalassemia will serve as a proof point, encouraging other developers to invest in new treatments for rare conditions in small patient populations, for example. Indeed, the rise of CRISPR has been rapid – there are currently more than 100 clinical trials underway using this technology, among other gene editing tools.
Advances in CRISPR clinical applications could also pave the way for treatments (or even cures) for more common diseases, such as multiple myeloma and type 1 diabetes. And the playbook can only be strengthened with new gene editing solutions; for example, most gene editing treatments in development are based on double strand DNA cutting, but new technologies – such as base and prime editing – are looking to modify the genome without double strand breaks thereby reducing the chance of unwanted DNA damage.
Though the approval of Casgevy brings hope to millions of people, true success will come when patients worldwide can access and benefit from these treatments. One way to accomplish this is by exploring different development and manufacturing processes that can help reduce costs and bring discoveries from bench to bedside more quickly. For instance, shifting to an allogeneic process, where “off the shelf” therapies are derived from a common donor to treat multiple patients, could help reduce the high expense and long timelines associated with autologous processes. Further down the road, we may even see the industry working to find a balance between the two processes to help reduce costs, decrease timelines, and simplify manufacturing where possible.
When it comes to the future of CRISPR, the road to progress will always have its twists and turns. The important thing to remember is that each step takes us closer to solving today’s most critical challenges.
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